Drug Developed by Professor Hua for Curing SMA Was Approved by the FDA

Near the end of 2016, great news generated buzz from the other side of the world. On December 24, the U.S. Food and Drug Administration (FDA) approved that the drug Nusinersen (product name: Spinraza) for treating spinal muscular atrophy (SMA) could be launched into the market. This is the first drug approved for treating SMA. FDA's unusual rapid approval has brought a gift and ray of hope to SMA-stricken patients and all patients suffering rare diseases worldwide. In the research and development stage of the drug, Professor Hua Yimin at the Institute of Neuroscience, through great efforts and hard work, eventually yielded great contributions.  


In 2004, Professor Hua commenced cooperation with Professor Krainer from the Cold Spring Harbor Laboratory (CSHL) and Ionis Pharmaceuticals. They jointly researched and developed antisense oligonucleotide drugs for treating SMA. During 2007 and 2011, Professor Hua published a series of landmark research findings as the lead author in international well-known journals, including Nature, Genes & Development, American Journal of Human Genetics, and PLoS Biology. In 2015, Professor Hua cooperated with CSHL again. By analyzing the efficacy of Nusinersen in different therapeutic approaches, he further made significant breakthroughs in the research of SMA mechanisms, the findings of which were published in Genes & Development with Soochow University as the lead author. Nusinersen was previously known as ASO10-27 and ISIS-SMNRx. The research and development of Nusinersen started in 2004, and the clinical trial of Nusinersen was launched globally in 2011. In 2006, Nusinersen was approved by the FDA, which took 12 years. Professor Hua is one of three co-inventors of the relevant drug patent.